Interest of luspatercept in treatment of anemia in lower-risk of myelodysplastic syndrome anemia: a single center study of 26 patients

Abstract

Myelodysplastic syndromes (MDS) are heterogeneous clonal hematological disorders characterized by ineffective hematopoiesis resulting in peripheral cytopenias. Luspatercept, an erythroid maturation agent, represents a novel therapeutic option for the management of anemia associated with low-risk MDS, particularly in patients with ring sideroblasts and/or SF3B1 mutations. We retrospectively studied 26 adults with low- or intermediate-risk MDS who were treated with luspatercept from 2021 to 2024 at the Nice University Hospital. Data were collected from ORBIS and CLINICOM and analyzed using Microsoft Excel. Treatment was initiated at 1 mg/kg, depending on the patient. The median age was 74 years, and 38.5% of patients had the SF3B1 mutation. Erythroid response was achieved in 53.8% of patients according to International Working Group (IWG) 2023. Treatment was generally well tolerated, with fatigue being the most common side effect (26.9%). Luspatercept appears to be effective in improving red blood cell production and reducing transfusion requirements in low-risk patients with MDS, with a favorable safety profile.

The authors' abstract, as published at the source. Blood Research, 2026 · DOI ↗